NZ597071A - Compositions and methods for modulation of smn2 splicing in a subject - Google Patents

Compositions and methods for modulation of smn2 splicing in a subject

Info

Publication number
NZ597071A
NZ597071A NZ597071A NZ59707110A NZ597071A NZ 597071 A NZ597071 A NZ 597071A NZ 597071 A NZ597071 A NZ 597071A NZ 59707110 A NZ59707110 A NZ 59707110A NZ 597071 A NZ597071 A NZ 597071A
Authority
NZ
New Zealand
Prior art keywords
sugar moiety
subject
medicament
smn2
antisense oligonucleotide
Prior art date
Application number
NZ597071A
Inventor
C Frank Bennett
Gene Hung
Frank Rigo
Adrian R Krainer
Yimin Hua
Marco A Passini
Lamya Shihabuddin
Seng H Cheng
Katherine W Klinger
Original Assignee
Isis Pharmaceuticals Inc
Cold Spring Harbor Lab
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=43356760&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=NZ597071(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Isis Pharmaceuticals Inc, Cold Spring Harbor Lab filed Critical Isis Pharmaceuticals Inc
Priority to NZ624712A priority Critical patent/NZ624712A/en
Publication of NZ597071A publication Critical patent/NZ597071A/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Abstract

Disclosed is the use of an antisense compound comprising an antisense oligonucleotide complementary to intron 7 of a nucleic acid encoding human SMN2 pre-mRNA, wherein each nucleoside of the antisense oligonucleotide comprises a modified sugar moiety, wherein each modified sugar moiety is a 2’-methoxyethyl sugar moiety, in the manufacture of a medicament for use in treating a disease or condition associated with survival motor neuron 1 (SMN1) in a subject, wherein the medicament is formulated for administration into the cerebrospinal fluid. Further disclosed is the use of an antisense compound comprising an antisense oligonucleotide complementary to intron 7 of a nucleic acid encoding human SMN2, wherein each nucleoside of the antisense oligonucleotide comprises a modified sugar moiety, wherein each modified sugar moiety is a 2’-methoxyethyl sugar moiety, in the manufacture of a medicament for treating a disease or condition associated with survival motor neuron 1 (SMN1) in a subject, wherein the medicament is formulated for increasing inclusion of exon 7 of SMN2 mRNA or amino acids in a SMN2 polypeptide in a motoneuron in a subject.
NZ597071A 2009-06-17 2010-06-17 Compositions and methods for modulation of smn2 splicing in a subject NZ597071A (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
NZ624712A NZ624712A (en) 2009-06-17 2010-06-17 Compositions and methods for modulation of smn2 splicing in a subject

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US21803109P 2009-06-17 2009-06-17
PCT/US2010/039077 WO2010148249A1 (en) 2009-06-17 2010-06-17 Compositions and methods for modulation of smn2 splicing in a subject

Publications (1)

Publication Number Publication Date
NZ597071A true NZ597071A (en) 2014-05-30

Family

ID=43356760

Family Applications (2)

Application Number Title Priority Date Filing Date
NZ597071A NZ597071A (en) 2009-06-17 2010-06-17 Compositions and methods for modulation of smn2 splicing in a subject
NZ624712A NZ624712A (en) 2009-06-17 2010-06-17 Compositions and methods for modulation of smn2 splicing in a subject

Family Applications After (1)

Application Number Title Priority Date Filing Date
NZ624712A NZ624712A (en) 2009-06-17 2010-06-17 Compositions and methods for modulation of smn2 splicing in a subject

Country Status (24)

Country Link
US (4) US8980853B2 (en)
EP (4) EP3643783A1 (en)
JP (7) JP5707396B2 (en)
KR (8) KR20220084437A (en)
CN (5) CN102665731A (en)
AU (2) AU2010262862C1 (en)
CA (1) CA2765396C (en)
CY (2) CY1120807T1 (en)
DK (2) DK3305302T3 (en)
ES (2) ES2699827T3 (en)
HK (1) HK1246648B (en)
HR (2) HRP20181712T1 (en)
HU (2) HUE039741T2 (en)
IL (2) IL217014A (en)
LT (2) LT3305302T (en)
MX (3) MX361732B (en)
NO (1) NO2020008I1 (en)
NZ (2) NZ597071A (en)
PL (2) PL3449926T3 (en)
PT (2) PT3449926T (en)
RU (2) RU2566724C9 (en)
SI (2) SI3449926T1 (en)
TR (1) TR201816256T4 (en)
WO (1) WO2010148249A1 (en)

Families Citing this family (106)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
PL2548560T3 (en) 2005-06-23 2015-11-30 Biogen Ma Inc Compositions and methods for modulation of SMN2 splicing
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
KR101881596B1 (en) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 Method for the synthesis of phosphorous atom modified nucleic acids
NZ597071A (en) 2009-06-17 2014-05-30 Isis Pharmaceuticals Inc Compositions and methods for modulation of smn2 splicing in a subject
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
AU2011257980B2 (en) 2010-05-28 2016-06-30 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
TWI541024B (en) 2010-09-01 2016-07-11 日本新藥股份有限公司 Antisense nucleic acid
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
WO2012039448A1 (en) 2010-09-24 2012-03-29 株式会社キラルジェン Asymmetric auxiliary group
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
KR102339196B1 (en) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 Peptide Oligonucleotide Conjugates
US9833439B2 (en) 2011-05-25 2017-12-05 Universite Paris Descartes ERK inhibitors for use in treating spinal muscular atrophy
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
EA201400566A1 (en) 2011-11-11 2014-09-30 Сэнтерис Фарма А/С CONNECTIONS INTENDED FOR MODELING SPLICING SMN2
KR102271212B1 (en) 2011-11-18 2021-07-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 Functionally-modified oligonucleotides and subunits thereof
AU2012345638C1 (en) 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
AU2012347765B2 (en) * 2011-12-06 2017-05-04 Research Institute At Nationwide Childrens's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
EP2872485B1 (en) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Asymmetric auxiliary group
KR102213609B1 (en) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 Chiral control
US9926559B2 (en) 2013-01-09 2018-03-27 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
WO2014113540A1 (en) * 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
BR112015022156A2 (en) 2013-03-14 2017-11-14 Isis Pharmaceuticals Inc compositions and methods for modulating tau expression
WO2014169243A2 (en) * 2013-04-12 2014-10-16 The Curators Of The University Of Missouri Smn2 element 1 antisense compositions and methods and uses thereof
TWI657819B (en) 2013-07-19 2019-05-01 美商Ionis製藥公司 Compositions for modulating tau expression
US10894963B2 (en) 2013-07-25 2021-01-19 Exicure, Inc. Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
US10568898B2 (en) 2013-08-13 2020-02-25 Northwestern University Lipophilic nanoparticles for drug delivery
JP2016531570A (en) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド Oligonucleotides targeting the euchromatin region
CA2930859C (en) 2013-09-04 2022-05-03 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
US20150197534A1 (en) 2013-09-05 2015-07-16 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
JPWO2015108048A1 (en) 2014-01-15 2017-03-23 株式会社新日本科学 Chiral nucleic acid adjuvant and antitumor agent having antitumor activity
WO2015108047A1 (en) 2014-01-15 2015-07-23 株式会社新日本科学 Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
AU2015207773B2 (en) 2014-01-16 2021-06-17 Wave Life Sciences Ltd. Chiral design
EP4162940A1 (en) 2014-04-17 2023-04-12 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN104163871A (en) * 2014-08-12 2014-11-26 大连医科大学 Anti-tumor specific splicing factor and preparation method thereof
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
US9976143B2 (en) 2014-10-03 2018-05-22 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016085986A1 (en) 2014-11-24 2016-06-02 Northwestern University High density lipoprptein nanoparticles for inflammation
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
MA41795A (en) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc EXCLUSION OF AN EXON INDUCED BY ANTISENSE COMPOUNDS IN MYOSTATIN
US10851371B2 (en) 2015-04-10 2020-12-01 Ionis Pharmaceuticals, Inc. Modulation of SMN expression
EP3851531A1 (en) 2015-06-01 2021-07-21 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type vii collagen
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
JP6987041B2 (en) * 2015-08-28 2021-12-22 サレプタ セラピューティクス，インコーポレイテッド Modified antisense oligomers for exon inclusion in spinal muscular atrophy
JP2018530560A (en) 2015-10-09 2018-10-18 サレプタ セラピューティクス， インコーポレイテッド Compositions and methods for the treatment of Duchenne muscular dystrophy and related disorders
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
EP3377073A4 (en) 2015-11-16 2019-06-26 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein
JP2019500345A (en) 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー Compositions and methods for the treatment of liver disease
CA3005254A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
EP3389725B1 (en) 2015-12-14 2022-04-06 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
KR102522059B1 (en) 2016-04-18 2023-04-14 사렙타 쎄러퓨틱스 인코퍼레이티드 Antisense oligomers and methods of their use to treat diseases associated with the acid alpha-glucosidase gene
WO2017193087A1 (en) 2016-05-06 2017-11-09 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs prsenting antisense oligonucleotides(aso) for specific knockdown of interleukin 17 receptor mrna
CN109641928B (en) 2016-06-14 2022-08-30 比奥根Ma公司 Hydrophobic interaction chromatography for oligonucleotide purification
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
KR20190020743A (en) 2016-06-24 2019-03-04 바이오젠 엠에이 인코포레이티드 Synthesis of Thiolated Oligonucleotides Without Capping Step
US11364304B2 (en) 2016-08-25 2022-06-21 Northwestern University Crosslinked micellar spherical nucleic acids
JOP20190065A1 (en) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing tau expression
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
FI3589730T3 (en) 2017-02-28 2024-02-22 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190200A1 (en) 2017-02-28 2019-08-27 Univ Pennsylvania Compositions useful in treatment of spinal muscular atrophy
CN110536964A (en) 2017-03-10 2019-12-03 国立研究开发法人国立成育医疗研究中心 Antisense oligonucleotides and the prevention of glycogen storage disease Ia type or therapeutic composition
JP2020517638A (en) 2017-04-20 2020-06-18 エータイアー ファーマ， インコーポレイテッド Compositions and methods for treating lung inflammation
US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
CN110753730A (en) 2017-06-19 2020-02-04 住友金属矿山株式会社 Near-infrared-curable ink composition, method for producing same, near-infrared-curable film, and stereolithography method
EP3652187B1 (en) 2017-07-10 2021-09-15 Geron Corporation Improved process for preparing imetelstat
BR122020005073A2 (en) 2017-08-04 2020-10-13 Skyhawk Therapeutics, Inc. COMPOUND, PHARMACEUTICAL COMPOSITION AND USE OF THE COMPOUND
CN111278991B (en) 2017-08-25 2022-04-01 斯托克制药公司 Antisense oligomers for the treatment of conditions and diseases
SG11202001783YA (en) 2017-10-12 2020-03-30 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
WO2019079637A2 (en) 2017-10-18 2019-04-25 Sarepta Therapeutics, Inc. Antisense oligomer compounds
US20210317474A1 (en) 2017-11-08 2021-10-14 Novartis Ag Means and method for producing and purifying viral vectors
EP3737755A1 (en) * 2018-01-12 2020-11-18 Camena Bioscience Limited Compositions and methods for template-free geometric enzymatic nucleic acid synthesis
US11447775B2 (en) 2018-01-12 2022-09-20 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
EP3743728A1 (en) 2018-01-25 2020-12-02 Biogen MA Inc. Methods of treating spinal muscular atrophy
WO2019168558A1 (en) * 2018-02-28 2019-09-06 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors
US20210002640A1 (en) * 2018-02-28 2021-01-07 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors
KR20210130854A (en) 2018-03-09 2021-11-01 다이이찌 산쿄 가부시키가이샤 Therapeutic agent for glycogen storage disease type ia
WO2019181946A1 (en) 2018-03-19 2019-09-26 国立大学法人東京医科歯科大学 Nucleic acid with reduced toxicity
KR20210018919A (en) 2018-06-08 2021-02-18 노파르티스 아게 Cell-based assays to measure drug product efficacy
CR20210050A (en) 2018-06-27 2021-06-10 Reborna Biosciences Inc Prophylactic or therapeutic agent for spinal muscular atrophy
JP2021534154A (en) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. Combination therapy for spinal muscular atrophy
MA54070A (en) 2018-10-29 2021-09-08 Biogen Ma Inc HUMANIZED AND STABILIZED FC5 VARIANTS FOR ENHANCED TRANSPORT ACROSS THE BLOOD-BRAIN BARRIER
TW202039859A (en) 2018-11-30 2020-11-01 美商艾夫西斯公司 Aav viral vectors and uses thereof
EP3918069A1 (en) 2019-01-31 2021-12-08 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
KR20210135241A (en) 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 Methods and compositions for controlling splicing
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
EP3942049A4 (en) 2019-03-20 2023-10-18 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
BR112021022219A2 (en) 2019-05-08 2022-03-22 Biogen Ma Inc Convergent liquid phase oligonucleotide synthesis
JP2022544538A (en) * 2019-08-15 2022-10-19 バイオジェン・エムエイ・インコーポレイテッド Combination therapy for spinal muscular atrophy
RU2738093C9 (en) * 2019-12-23 2020-12-29 Общество с ограниченной ответственностью "Гелеспон" Synthesis method of 2'-o-(2-methoxyethyl) thiophosphate oligonucleotide
US20220064638A1 (en) 2020-02-28 2022-03-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
CN113444722A (en) * 2020-03-24 2021-09-28 中国科学院脑科学与智能技术卓越创新中心 Application of single base editing mediated splicing repair in preparation of drugs for treating spinal muscular atrophy
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
IL300287A (en) * 2020-07-31 2023-04-01 Ractigen Therapeutics Combinatory treatment of sma with sarna and mrna modulators
AR125239A1 (en) 2021-04-02 2023-06-28 Kumiai Chemical Industry Co HETEROCYCLIC COMPOUND AND USE THEREOF
WO2023073526A1 (en) 2021-10-25 2023-05-04 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
WO2023117965A1 (en) 2021-12-20 2023-06-29 Freie Universität Berlin Methods and agents for increasing rbm3 expression
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders

Family Cites Families (125)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5927900A (en) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk Oligonucleotide derivative and its preparation
FR2540122B1 (en) 1983-01-27 1985-11-29 Centre Nat Rech Scient NOVEL COMPOUNDS COMPRISING A SEQUENCE OF OLIGONUCLEOTIDE LINKED TO AN INTERCALATION AGENT, THEIR SYNTHESIS PROCESS AND THEIR APPLICATION
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (en) 1984-07-19 1989-02-17 Centre Nat Rech Scient NOVEL OLIGONUCLEOTIDES, THEIR PREPARATION PROCESS AND THEIR APPLICATIONS AS MEDIATORS IN DEVELOPING THE EFFECTS OF INTERFERONS
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (en) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd Poly-labeled oligonucleotide derivative
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (en) 1987-11-12 1989-05-24 Max Planck Gesellschaft MODIFIED OLIGONUCLEOTIDS
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
FR2645345A1 (en) 1989-03-31 1990-10-05 Thomson Csf METHOD FOR DIRECT MODULATION OF THE COMPOSITION OR DOPING OF SEMICONDUCTORS, IN PARTICULAR FOR THE PRODUCTION OF ELECTRONIC MONOLITHIC COMPONENTS OF THE PLANAR TYPE, USE AND CORRESPONDING PRODUCTS
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
EP0942000B1 (en) 1989-10-24 2004-06-23 Isis Pharmaceuticals, Inc. 2'-Modified oligonucleotides
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ES2116977T3 (en) 1990-05-11 1998-08-01 Microprobe Corp SOLID SUPPORTS FOR NUCLEIC ACID HYBRIDIZATION TESTS AND METHODS TO IMMOBILIZE OLIGONUCLEOTIDES IN A COVALENT WAY.
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
ATE198598T1 (en) 1990-11-08 2001-01-15 Hybridon Inc CONNECTION OF MULTIPLE REPORTER GROUPS ON SYNTHETIC OLIGONUCLEOTIDES
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
DE59208572D1 (en) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclic nucleosides, oligonucleotides, processes for their preparation and intermediates
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
FR2687679B1 (en) 1992-02-05 1994-10-28 Centre Nat Rech Scient OLIGOTHIONUCLEOTIDES.
EP0577558A2 (en) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclic nucleosides having bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
CA2159631A1 (en) 1993-03-30 1994-10-13 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
DE4311944A1 (en) 1993-04-10 1994-10-13 Degussa Coated sodium percarbonate particles, process for their preparation and detergent, cleaning and bleaching compositions containing them
EP0698092B1 (en) 1993-05-11 2007-07-25 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
NZ282981A (en) 1994-02-28 1998-08-26 Agritech Lab Inc Increasing egg production in female birds by administering heterologous inhibin protein and a carrier protein
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
EP0708178A1 (en) * 1994-10-19 1996-04-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
EP1108724B1 (en) 1996-01-16 2007-09-19 Sirna Therpeutics, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
US7875733B2 (en) 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
KR100782896B1 (en) 1999-05-04 2007-12-06 엑시콘 에이/에스 L-Ribo-LNA analogues
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US6649411B2 (en) 1999-07-30 2003-11-18 The United States Of America As Represented By The Department Of Health And Human Services Methods of inhibiting cancer cells with ADNF III antisense oligonucleotides
US6770633B1 (en) 1999-10-26 2004-08-03 Immusol, Inc. Ribozyme therapy for the treatment of proliferative skin and eye diseases
US20050020525A1 (en) 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US6962906B2 (en) 2000-03-14 2005-11-08 Active Motif Oligonucleotide analogues, methods of synthesis and methods of use
AU2001259706A1 (en) 2000-05-09 2001-11-20 Reliable Biopharmaceutical, Inc. Polymeric compounds useful as prodrugs
ATE544473T1 (en) 2000-11-09 2012-02-15 Cold Spring Harbor Lab CHIMERIC MOLECULES FOR MODULATION OF GENE EXPRESSION
US6376508B1 (en) 2000-12-13 2002-04-23 Academia Sinica Treatments for spinal muscular atrophy
WO2003004602A2 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
GB0219143D0 (en) 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
JP2006507841A (en) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド Functional and ultrafunctional siRNA
JP2004344072A (en) * 2003-05-22 2004-12-09 Japan Science & Technology Agency Splicing control element for smn gene
WO2004113867A2 (en) 2003-06-16 2004-12-29 University Of Massachusetts Exon analysis
ES2382807T3 (en) 2003-08-28 2012-06-13 Takeshi Imanishi New artificial nucleic acids of the N-O link type with cross-linking
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
GB0425625D0 (en) * 2004-11-22 2004-12-22 Royal College Of Surgeons Ie Treatment of disease
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
PL2548560T3 (en) 2005-06-23 2015-11-30 Biogen Ma Inc Compositions and methods for modulation of SMN2 splicing
JP4223027B2 (en) 2005-06-30 2009-02-12 シャープ株式会社 Image forming apparatus and secret data transmission method
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
KR101789603B1 (en) 2005-11-10 2017-11-21 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
EP3210633B1 (en) * 2006-01-26 2019-06-19 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
US8129515B2 (en) 2006-01-27 2012-03-06 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
KR20130042043A (en) 2006-01-27 2013-04-25 아이시스 파마수티컬즈 인코포레이티드 6-modified bicyclic nucleic acid analogs
DK2066684T3 (en) 2006-05-11 2012-10-22 Isis Pharmaceuticals Inc 5'-Modified Bicyclic Nucleic Acid Analogs
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009120700A2 (en) 2008-03-26 2009-10-01 Families Of Spinal Muscular Atrophy Inhibition of dcps
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
WO2010123594A2 (en) 2009-01-15 2010-10-28 Children's Medical Center Corporation Device for filtration of fluids there through and accompanying method
US20120021515A1 (en) 2009-02-06 2012-01-26 Swayze Eric E Oligomeric compounds and methods
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
NZ597071A (en) 2009-06-17 2014-05-30 Isis Pharmaceuticals Inc Compositions and methods for modulation of smn2 splicing in a subject
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
US8183002B2 (en) 2009-12-03 2012-05-22 Abbott Laboratories Autoantibody enhanced immunoassays and kits
US10653713B2 (en) 2010-10-06 2020-05-19 Medtronic, Inc. Methods for distributing agents to areas of brain
MX2014000480A (en) 2011-07-13 2014-06-23 Merck Sharp & Dohme Method for detection of amyloid beta oligomers in a fluid sample and uses thereof.
US9926559B2 (en) 2013-01-09 2018-03-27 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
US20140367278A1 (en) 2013-06-12 2014-12-18 PharmOptima, LLC Methods for Detecting Survival Motor Neuron (SMN) Protein in Whole Blood or Cerebral Spinal Fluid

Also Published As

Publication number Publication date
EP2442816A1 (en) 2012-04-25
MX2018015635A (en) 2020-11-12
CY1122687T1 (en) 2021-03-12
JP2017061497A (en) 2017-03-30
DK3305302T3 (en) 2018-11-19
RU2683772C2 (en) 2019-04-02
HUE046966T2 (en) 2020-04-28
AU2010262862B2 (en) 2015-10-29
HRP20191978T1 (en) 2020-05-15
CN115227710A (en) 2022-10-25
JP6370860B2 (en) 2018-08-08
RU2566724C2 (en) 2015-10-27
CY1120807T1 (en) 2019-12-11
PL3449926T3 (en) 2020-06-01
JP6064139B2 (en) 2017-01-25
IL245096A0 (en) 2016-06-30
JP2022025131A (en) 2022-02-09
SI3305302T1 (en) 2018-12-31
AU2016200344B2 (en) 2017-12-21
AU2010262862C1 (en) 2020-04-30
AU2016200344A1 (en) 2016-02-11
US20170015995A1 (en) 2017-01-19
RU2015139553A (en) 2018-12-25
MX2011014004A (en) 2012-06-19
US8980853B2 (en) 2015-03-17
HUE039741T2 (en) 2019-01-28
JP5707396B2 (en) 2015-04-30
KR20170054537A (en) 2017-05-17
LT3305302T (en) 2018-12-10
CN116440150A (en) 2023-07-18
JP2020073498A (en) 2020-05-14
CN102665731A (en) 2012-09-12
JP2024012416A (en) 2024-01-30
MX361732B (en) 2018-12-14
KR101965868B1 (en) 2019-04-05
CN106983768B (en) 2023-04-07
US20190030058A1 (en) 2019-01-31
HK1246648B (en) 2019-09-06
US9717750B2 (en) 2017-08-01
JP2015131828A (en) 2015-07-23
KR20220084437A (en) 2022-06-21
US20200376018A1 (en) 2020-12-03
CA2765396A1 (en) 2010-12-23
EP3643783A1 (en) 2020-04-29
NZ624712A (en) 2015-10-30
CN115227709A (en) 2022-10-25
LT3449926T (en) 2020-01-27
KR20190017063A (en) 2019-02-19
SI3449926T1 (en) 2020-04-30
KR20240036132A (en) 2024-03-19
JP2012530715A (en) 2012-12-06
ES2699827T3 (en) 2019-02-13
IL217014A0 (en) 2012-02-29
PT3449926T (en) 2019-11-12
KR20190037378A (en) 2019-04-05
KR20210057223A (en) 2021-05-20
EP2442816A4 (en) 2013-10-02
EP3305302B1 (en) 2018-09-19
DK3449926T3 (en) 2019-11-11
RU2012101491A (en) 2013-07-27
RU2566724C9 (en) 2019-03-12
IL245096B (en) 2018-04-30
WO2010148249A1 (en) 2010-12-23
ES2761614T3 (en) 2020-05-20
EP3449926A1 (en) 2019-03-06
RU2015139553A3 (en) 2018-12-25
PL3305302T3 (en) 2019-02-28
US20120190728A1 (en) 2012-07-26
CN106983768A (en) 2017-07-28
EP3449926B1 (en) 2019-10-09
AU2010262862A1 (en) 2012-01-19
NO2020008I1 (en) 2020-04-01
KR20120093138A (en) 2012-08-22
EP3305302A1 (en) 2018-04-11
JP6637121B2 (en) 2020-01-29
IL217014A (en) 2016-04-21
CA2765396C (en) 2019-08-20
HRP20181712T1 (en) 2018-12-28
JP2018184423A (en) 2018-11-22
KR20200047790A (en) 2020-05-07
TR201816256T4 (en) 2018-11-21
PT3305302T (en) 2018-12-14

Similar Documents

Publication Publication Date Title
NZ597071A (en) Compositions and methods for modulation of smn2 splicing in a subject
US11077132B2 (en) Tau antisense oligomers and uses thereof
HRP20181030T1 (en) Serpina1 irna compositions and methods of use thereof
US20180023081A1 (en) Lna oligonucleotides with alternating flanks
SG171676A1 (en) Compositions and methods for inhibiting expression of the pcsk9 gene
JP2016506240A5 (en)
WO2013074974A3 (en) Modified rnai agents
SI2929031T1 (en) Pcsk9 irna compositions and methods of use thereof
JP2012530715A5 (en)
JP2016513976A5 (en)
WO2009090639A3 (en) Sirna compounds and methods of use thereof
WO2014022739A3 (en) Modified rnai agents
BR112015024729B1 (en) ANTISSENSE OLIGONUCLEOTIDE, ITS USE AND PHARMACEUTICAL COMPOSITION
JP2011500858A5 (en)
WO2009105260A3 (en) Ultra-small rnas as toll-like receptor-3 antagonists
WO2008036933A3 (en) Compositions and methods for inhibiting expression of the hamp gene
WO2008036638A3 (en) Rnai modulation of scap and therapeutic uses thereof
WO2007137156A8 (en) Rnai modulation of aha and therapeutic uses thereof
WO2008008719A3 (en) Compositions and methods for inhibiting expression of the myc gene
UA106209C2 (en) Choline cocrystal of 5-[(1z,2e)-2-methyl-3-phenylpropenylidene]-4-oxo-2-thioxo-3-thiazolidine acetic acid
EP3558328A1 (en) Compositions and methods for reprogramming somatic cells into induced vasculogenic cells
EP4328306A2 (en) Angelman syndrome antisense treatment
WO2011133584A3 (en) Nucleic acid compounds for inhibiting hras gene expression and uses thereof
AU2016312590B2 (en) Methods for treatment of polycystic kidney disease
ATE522540T1 (en) SHORT INTERFERING RIBONUCLEIC ACIDS FOR THE TREATMENT OF ALLERGY DISEASES

Legal Events

Date Code Title Description
ASS Change of ownership

Owner name: COLD SPRING HARBOR LABORATORY, US

Free format text: OLD OWNER(S): ISIS PHARMACEUTICALS, INC.; GENZYME CORPORATION; COLD SPRING HARBOR LABORATORY

Owner name: ISIS PHARMACEUTICALS, INC., US

Free format text: OLD OWNER(S): ISIS PHARMACEUTICALS, INC.; GENZYME CORPORATION; COLD SPRING HARBOR LABORATORY

PSEA Patent sealed
RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 3 YEARS UNTIL 17 JUN 2017 BY PIZZEYS PATENT AND TRADE MARK ATTORNEYS

Effective date: 20140903

ASS Change of ownership

Owner name: COLD SPRING HARBOR LABORATORY, US

Effective date: 20161124

Owner name: BIOGEN MA INC., US

Effective date: 20161124

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 17 JUN 2018 BY COMPUTER PACKAGES INC

Effective date: 20170531

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 17 JUN 2019 BY RENEWALS TEAM

Effective date: 20180525

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 17 JUN 2020 BY RENEWALS TEAM

Effective date: 20190611

S38C Proceedings under section 38 (amendment of specification with leave of commissioner): specification amended
S38C Proceedings under section 38 (amendment of specification with leave of commissioner): specification amended
RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 17 JUN 2021 BY CPA GLOBAL

Effective date: 20200430

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 17 JUN 2022 BY CPA GLOBAL

Effective date: 20210506

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 17 JUN 2023 BY CPA GLOBAL

Effective date: 20220505

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 17 JUN 2024 BY ANAQUA SERVICES

Effective date: 20230524